The FDA is investigating reports of tumors in patients previously treated with Novartis’ Zolgensma, a gene therapy for spinal muscular atrophy (SMA).
Novartis reported nine tumor cases among patients treated since Zolgensma’s 2019 approval. Tumors were reported in the kidneys, nervous system, and areas in or around the spine.
Three cases involved the cancer-associated gene GLI1. In two, testing found that DNA from the therapy’s AAV viral vector had integrated into the patient’s genome near or within GLI1. The third case is still being investigated.
The FDA says the potential risk also applies to Itvisma, another Novartis SMA therapy using the same vector and construct. It is assessing whether regulatory action is needed.
The FDA says the benefits of Zolgensma and Itvisma continue to outweigh their potential risks for approved uses while the review proceeds. The reports do not mean that every tumor was caused by treatment.
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